Purpose of the STSM: This STSM involved application of antisense oligonucleotides delivery as a substrate reduction therapy for patient cell lines of Mucopolysaccharidoses type III which is a type of lysosomal storage disorder. Substrate reduction therapy is a common approach to treat lysosomal storage disorders and in this project siRNA-mediated substrate reduction therapy was performed. The purpose of STSM was to expand our knowledge about delivery techniques of antisense RNAs to patient fibroblasts (in vitro studies) in order to use this technology as substrate reduction therapy. As in the Seyrantepe research group, we are working on a project with antisense therapies applied to in vitro cell cultures and animal models within the scope of CA17103 CostAction project. Thus it was very beneficial to learn some experimental and theoretical protocols and methods to work with delivery of antisense RNA to Tay-Sachs mouse model and patient cell lines in our laboratory.

Grantee name: Nurselin Ateş (…)
STSM start and end date: 2020-03-02 to 2020-03-13
Title: Delivery of in vitro Antisense RNA Therapeutics to Mucopolysaccharidosis III
Host lab: Research and Development Unit, Department of Human Genetics, INSA (Porto, Portugal)